Medical Marijuana and Cannabidiol (CBD)

In the last several years there has been renewed interest in the therapeutic potential of marijuana (cannabis and its derivatives) for myriad conditions including epilepsy, and specifically infantile spasms. The overwhelming source of enthusiasm has been from online parent testimonials and press reports of success in individual patients. Although the internet is replete with testimonials extolling the virtue of medical marijuana, there is insufficient evidence—and especially safety data—to recommend its use for treatment of seizures in most cases. That said, a series of clinical trials demonstrated that a pharmaceutical formulation of cannabidiol (Epidiolex®, GW Pharmaceuticals) is effective in the treatment of seizures associated with Lennox-Gastaut Syndrome (a form of epilepsy which often follows infantile spasms) as well as Dravet Sydrome (a form of epilepsy that is distinct from infantile spasms. These studies provided the basis for the United States Food and Drug Administration (FDA) approval of Epidiolex. Importantly, the effectiveness of Epidiolex has not been establsihed outside of these two specific forms of epilepsy. Also of note, a study on the abuse potential of cannabidiol (using Epidioelex) demonstrated that pure cannabidiol does not appear to be addictive and poses little risk of abuse. The FDA approval of Epidiolex and the aforementioned abuse study led the United States Drug Enforcement Agency (DEA) to reclassify Epidiolex (but not other form of cananbidiol) as a "Schedule 5" drug, indicating that there is an accepted medical use, and low potential for abuse. 

At this point, Epidiolex is legal in the United States, though its legal standing as a cannabis derivative is unclear in other countries. In the United States, most cannabidiol products--excluding hemp-derived cannabidiol—remain federally illegal, but in contradictory and confusing fashion—legal in many states under an array of medical marijuana provisions. An array of legal risks continue to surround the use and manufacture of most cannabidiol-rich products, excluding Epidiolex. Therefore, children, parents, "prescribing" physicians, and "manufacturers" (growers/distributors) of marijuana preparations in all states are potentially vulnerable to arrest and federal prosecution. Furthermore, the possession and administration of these products are officially forbidden in most hospitals because of DEA oversight and licensure. Having said this, we are unaware of any case in the United States in which a parent, health-care provider, or hospital has been prosecuted or faced other disciplinary action for using, administering, or recommending a marijuana-derived treatment in a good-faith effort to treat epilepsy.

With regard to any cannabidiol product, it is dangerous to try these therapies without informing your child's physicians, especially those who prescribe other drugs. Most readily available formulations of medical marijuana contain numerous—more than 100—chemicals derived from the plant, many of which may interact with commonly prescribed medications. There is compelling evidence of a strong interaction between cannabidiol (CBD) and clobazam (Onfi®, Frizium®), such that blood levels of an active metabolite (N-desmethyclobazam, or norclobazam) may rise considerably. Conversely, clobazam exposure appears to increase blood levels of an active cannabidiol metabolite (7-OH-cannabidiol). This effect on clobazam metabolism may explain favorable response in some patients, as well as some side effects that have been attributed to cannabidiol. 

There are efforts underway to rigorously evaluate the potential usefulness of medical marijuana for treatment of infantile spasms. The following reports reflect a sampling of studies which suggest—but do not establish—effectiveness and safety:

Potential efficacy of cannabidiol for treatment of refractory infantile spasms and Lennox-Gastaut syndrome

Authors: Raymond Zhou, Catherine Jacobson, Julius Weng, Emily Cheng, Johnson Lay, Phoebe Hung, Jason Lerner, Raman Sankar and Shaun Hussain

This study—conducted by UCLA investigators who manage this website—is a presentation of an online survey of parents who have administered CBD-enriched cannabis preparations to their children for the treatment of severe epilepsy, including many patients with infantile spasms. Among 200 respondents, 117 had actually administered CBD products to their children. 53 children suffered from infantile spasms and/or Lennox Gastaut syndrome. Over 90% reported reductions in seizure frequency, and 13% reported seizure-freedom. Reported side effects were modest. Among the minority of parents who were able and willing to report exact CBD dosage, the typical dose was 4 mg/kg/day (1.8 mg/pound/day). Dosage was not different among children with and without improvement.

Cannabidiol treatment of refractory epileptic spasms: an open label study

Authors: Elena Abati, Evan Hess, Amy Morgan, Patricia L. Bruno, Elizabeth Thiele

In this report, Dr. Abati and colleagues report on the response of 9 patients with epileptic spasms (infantile spasms persisting well into childhood) to purified cannabidiol in a small open-label study. 30-50% of children had at least a 50% reduction in burden of seizures at 3 to 12 months, and 2 children were seizure-free at 2 months.

A phase-2 study of pharmaceutical grade synthetic cannabidiol oral solution for treatment of refractory infantile spasms

Authors: Shaun Hussain, Dennis Dlugos, M. Roberta Cilio, Neha Parikh, Alexander Oh, Raman Sankar

In this study Dr. Hussain and colleagues treated 9 patients with infantile spasms who had previously failed treatment with ACTH and vigabatrin. Although 8 of 9 patients exhibited no response, one patient exhibited rapid resolution of both hypsarrhythmia and seizures (infantile spasms). This sole responder relapsed shortly thereafter. Given the severity and duration of infantile spasms in this cohort, this modest result suggests that cannabidiol may nevertheless be effective, especially in less refractory populations. Further study is clearly warranted, and this study certainly does not prove that cannabidiol is (or is not) effective and safe in the treatment of infantile spasms.


These studies do not provide adequate evidence to conclude that pure CBD or CBD-enriched cannabis extracts are safe and effective for treatment of infantile spasms. All investigators highlighted the need for well-designed clinical trials to properly evaluate the effectiveness and safety of these products. These studies demonstrated wide discrepancies in response rate, and none were able to control for the impact of bias (e.g. placebo-like effects). Despite the favorable response rate in the study and Zhou and colleagues, it is still quite possible that CBD is ineffective and unsafe. The fantastically high response rates observed by Zhou et al and the observation of Press et al that patients who moved to Colorado were substantially more likely to report benefit suggests that response rates are highly vulnerable to bias; at this point all response rates to date should be interpreted with extreme caution. At present, a clinical trial evaluating the first-line use of pure synthetic cannabidiol is underway ( NCT033421496).


Although efforts are made to keep this website correct and up-to-date, we urge caution in interpreting the information you find here. This is in no way a substitute for the advice and care of a pediatric neurologist. Please view the terms of use.

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